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Перспективы применения некодирующих РНК в генной терапии (обзор)
Аннотация
В обзоре освещены возможности применения некодирующих РНК, действующих по механизму РНК-интерференции. Проанализированы особенности строения, способы повышения стабильности малых интерферирующих РНК, а также методы усиления эффективности посттранскрипционного сайленсинга. Описаны некоторые методы химической модификации олигонуклеотидов и системы их доставки в клетку. Рассмотрены новейшие лекарственные препараты на основе некодирующих РНК. Описаны их фармакокинетика и фармакодинамика, ход клинических исследований. Особое внимание уделено биобезопасности данных препаратов.
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